By Barry Springer, Ph.D., Vice President and Head of Biologics Strategy and External Innovation, Janssen BioTherapeutics
The biologics space is rapidly expanding beyond just antibodies. Hot new technologies, ranging from gene therapy, immunotherapies, CRISPR and CAR T-cell therapies as well as next-generation antibodies such as bispecifics, continue to drive growth in this space. Venture capital investment has responded in turn and is shifting towards biologics, with the share of biologics R&D funded by venture capital rising from 27 percent in 2004 to 50 percent in 2013. Currently, biologics are on track to be 20 percent of total market sales by 2017.
This anticipated market growth makes biologics an extremely attractive market for startups, which translates into an increase in the number of biologic Investigational New Drug (IND) applications submitted. However, submitting a biologic IND can be a tricky process, especially if you’ve never done it before, and you may have questions, such as:
- What should I consider when designing a preclinical toxicology program? Does this differ for a small molecule versus an antibody?
- How do I select the optimal first-in-human dosage?
- What is an immunogenicity risk assessment and why is it important? What is included in this document?
- Should I submit the IND to the Center for Biologics Evaluation and Research (CBER) or the Center for Drug Evaluation and Research (CDER)?
- What is discussed in the pre-pre IND meeting? What about the pre-IND meeting?
With potential investors and pharma partners seeking strong strategic IND plans during due diligence, it is important to make sure you have the right strategy in place, which will save you time, money and a lot of stress down the line. Finding someone who has that “been there, done that” experience may be difficult, but it’s essential for entrepreneurs to get answers to these and other questions--and ideally from someone who has done this before. Seek out individuals that have gone through the process successfully and learn as much as you can about the processes, procedures and best practices for filing a biologic IND. Have the tough discussions with them and learn what worked and what didn’t, and don’t be afraid of the frank answers you may hear. It’s all part of the learning process.
These discussions can bring into focus the specific steps that a company can take to prepare for IND filing. For example, I recall a conversation I had with the CEO of an RNA therapeutics company while sharing a cab crossing the Charles River after a JLABS event. After hearing more details about the company’s technology, it became clear to me that the company needed to conduct primate studies, in addition to the mouse studies that were planned, in order to adequately validate the technology prior to IND filing. Based on our discussion, the CEO implemented this plan, and now laughs when he recalls this “conversation over the river” that had such an impact on the company. You can also tap resources within larger pharma companies with biologics experts in their Research & Development departments to get their opinion on what constitutes success.
If you are preparing to file a biologic IND and would like insight on crafting a strategy, or are just interested in learning about the process in general, please feel free to contact me:
Barry Springer, Ph.D., Vice President and Head of Biologics Strategy and External Innovation, Janssen BioTherapeutics BSPRINGE@its.jnj.com